NEJM This Week NEJM This Week — May 1, 2025
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Apr 30, 2025 Explore the impact of high lipoprotein(a) on heart disease and innovative RNA therapies like lepodicerin. Discover groundbreaking lentiviral gene therapy for severe immune disorders and its accessibility challenges. Dive into COPD management with mepolizumab, which shows promise but lacks quality-of-life benefits. Delve into the serious health risks of alcohol consumption and potential policy solutions. Finally, hear an inspiring story of resilience and camaraderie between a cancer patient and his doctor.
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Lepodicerin's Impact on Lipoprotein(a)
- Lepodicerin significantly reduces lipoprotein(a) levels with a long-duration effect exceeding other agents.
- Despite high risk, less than 1% of Americans with elevated lipoprotein(a) have been tested clinically.
EPO Variants in Polycythemia
- Secondary erythrocytosis can arise from non-coding EPO variants producing more active hepatic-like EPO.
- Non-coding regions of EPO require screening to understand and potentially treat erythrocytosis genetically.
Gene Therapy for Leukocyte Adhesion Deficiency
- Lentiviral gene therapy for LAD1 shows 100% survival without need for allogeneic transplant.
- Challenges remain to ensure these costly, complex therapies are accessible to vulnerable patients.